RARECast

512 Episodes

1. Why Reforms to the Accelerated Approval Pathway Threaten Rare Disease Drug Development

   Published: 6/30/2022
2. A Longstanding Academic-Nonprofit Collaboration Gives Rise to an ALS Drug Company

   Published: 6/16/2022
3. Targeting Regulatory RNA to Upregulate Gene Expression to Treat Rare Diseases

   Published: 6/9/2022
4. Developing a New Approach to Treat Rare, Autoimmune Conditions

   Published: 6/2/2022
5. Bridging the Gap Between Basic and Commercial Research for Rare Disease

   Published: 5/26/2022
6. Stoking Functional Copies of Genes to Compensate for Mutated Ones

   Published: 5/19/2022
7. Targeting ALS with Synergistic Combinations of Therapies

   Published: 5/13/2022
8. How a Polish Rare Disease Organizations Is Helping Its Ukrainian Neighbors

   Published: 5/5/2022
9. Powering a New Era of Genetic Medicine

   Published: 4/28/2022
10. Searching for a Diagnosis

    Published: 4/21/2022
11. A Novel Way to Deliver Rare Disease Therapies

    Published: 4/14/2022
12. Bringing Diversity, Equity, and Inclusion to the Books We Read

    Published: 4/7/2022
13. From Mila to Millions: Scaling N of 1 Therapies

    Published: 3/31/2022
14. Genetically Modifying Patients’ Skin Cells to Treat Rare Diseases

    Published: 3/24/2022
15. Targeting Rare, Immunologic Disorders

    Published: 3/18/2022
16. Harnessing a Natural Mechanism to Silence Disease

    Published: 3/10/2022
17. Advancing Precision Medicine with Patient Data

    Published: 3/3/2022
18. Pregnancy and Motherhood for a Woman with SMA

    Published: 2/24/2022
19. Steps to Improve Data Gathering of Rare Diseases

    Published: 2/17/2022
20. Rare Patients Learn to Make Their Voices Heard

    Published: 2/10/2022